发明名称 |
Compositions and methods for inhibiting expression of a mutant gene |
摘要 |
The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.
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申请公布号 |
US7763590(B2) |
申请公布日期 |
2010.07.27 |
申请号 |
US20030384463 |
申请日期 |
2003.03.07 |
申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
KREUTZER ROLAND;LIMMER STEFAN;JOHN MATTHIAS |
分类号 |
A61K48/00;A61K38/00;A61P35/00;C12N7/01;C12N15/113 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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